Hologene 5    (DrugBank: -)

1 disease
IDDisease name (Link within this page)Number of trials
36Epidermolysis bullosa1

36. Epidermolysis bullosa    [ 147 clinical trials,   170 drugs,   (DrugBank: 40 drugs),   32 drug target genes,   113 drug target pathways]
Searched query = "Epidermolysis bullosa", "EBS", "JEB", "DDEB", "RDEB", "Kindler syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 147 trial found
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1EUCTR2018-000261-36-IT
(EUCTR)
07/10/2020Gene Therapy for patient with Junctional Epidermolysis BullosaMULTICENTRE, OPEN-LABEL, UNCONTROLLED, PIVOTAL CLINICAL TRIAL TO CONFIRM THE EFFICACY AND SAFETY OF AUTOLOGOUS FIBRIN-CULTURED EPIDERMAL GRAFTS CONTAINING EPIDERMAL STEM CELLS GENETICALLY MODIFIED FOR RESTORATION OF EPIDERMIS IN PATIENTS WITH JUNCTIONAL EPIDERMOLYSIS BULLOSA (HOLOGENE 5) - Hologene 5 Inherited Epidermolysis Bullosa (EB) is a group of rare, devastating genetic disorders characterized by structural and mechanical fragility ofskin and mucosal membranes, impairing the patient's quality of life. Generalized JEB is a chronic, life-threatening condition caused bymutations in genes– encoding different chains of laminin 332. All of these mutations hamper hemidesmosome formation, causing blisters. The most frequent, and perhaps most severe, JEB is due to mutations in LAMB3.
MedDRA version: 20.0;Level: PT;Classification code 10014989;Term: Epidermolysis bullosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]
Product Name: Hologene 5
Product Code: [Hologene 5 DS]
Other descriptive name: Ex-vivo expanded autologous human keratinocytes suspension containing epidermal stem cells genetically modified with a gamma-retroviral (rv) vector expressing the full-length LAMB3 cDNA.
HOLOSTEM TERAPIE AVANZATE S.R.L.NULLNAFemale: yes
Male: yes
6Phase 2;Phase 3France;Germany;Italy